Pharmaceutical Technology

SEPTEMBER 30, 2022

Skysona is indicated as a one-time gene therapy to slow the progression of cerebral adrenoleukodystrophy (CALD), a rare paediatric neurodegenerative disease in boys aged 4–17 years diagnosed with early-stage CALD. Prior to bluebird's approvals, there were only two FDA-approved gene therapies for inherited conditions on the market.

FDA 115

FDA Insurance 115

pharmaphorum

JUNE 12, 2022

Thursday saw the Cellular, Tissue and Gene Therapies advisory committee back elivaldogene autotemcel (eli-cel) for cerebral adrenoleukodystrophy (CALD) – see our report here – and the following day it was the turn of betibeglogene autotemcel (beti-cel) for beta thalassaemia.

FDA 58

FDA 58

pharmaphorum

AUGUST 18, 2022

million price tag attached to the therapy when it was first made available in Europe, and is also well above the proposed $2.1 Zynteglo is a customised, one-time treatment created using a patient’s own bone marrow stem cells hat are genetically modified to produce functional beta-globin, which is mutated in the disease.

FDA 52

FDA Insurance 52

Pharmaceutical Technology

JUNE 29, 2022

The US Food and Drug Administration (FDA) put a high-profile bluebird bio trial for sickle cell disease on partial clinical hold, and advisory panels deliberated over decisions involving gene therapies for amyotrophic lateral sclerosis (ALS), cerebral adrenoleukodystrophy (CALD), and beta-thalassemia.

Pharmaceutical Companies 98

Pharmaceutical Companies Pharmaceutical Companies FDA 98